Sequential Transplantation of UCBSCs and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency T1DM

Sequential Transplantation of Umbilical Cord Blood Stem Cells and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency Type 1 Diabetes Mellitus

This study evaluates the efficacy of sequential transplantation of umbilical cord blood stem cells and islet cells in children with monogenic immunodeficiency type 1 diabetes mellitus. Umbilical cord blood stem cell transplantation will be performed first. Children with stable immune reconstruction will than receive islet cell transplantation.

Study Overview

• Status: Recruiting
• Conditions: Immunologic Deficiency Syndromes; Diabetes Mellitus, Type 1
• Intervention / Treatment: Procedure: Sequential transplantation

Detailed Description

Monogenic immunodeficiency type 1 diabetes mellitus (T1DM) usually onsets in early age and has a long course of treatment. Because of T cell deficiency, patients are prone to recurrent infection, hemorrhage, sepsis, colitis or complications of diabetes mellitus, which lead to early death.

New clinical treatment schemes have been explored and introduced around the world. Sequential transplantation of umbilical cord blood stem cells and islet cells is the latest treatment method for these children. Early treatment of monogenic immunodeficiency T1DM children can avoid disease-related organ toxicity, infection risk associated with chronic immunosuppression, and possible prevention of autoimmune endocrine organ damage. Thus, sequential transplantation of umbilical cord blood stem cells and islet cells is the only possible cure for those patients currently.

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Luo Feihong; Phone Number: +862164931226 +862164931226; Email: luofh@fudan.edu.cn
• Study Contact Backup: Name: Xu Zhenran; Phone Number: +862164931226 +862164931226; Email: xu_zhenran@163.com

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China
      • Recruiting
      • Children's Hospital of Fudan University
      • Contact: Luo Feihong; Phone Number: +862164931226 +862164931226; Email: luofh@fudan.edu.cn
      • Contact: Xu Zhenran; Phone Number: +862164931226 +862164931226; Email: xu_zhenran@fudan.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1.Type 1 diabetes mellitus children with genetic immunodeficiency

1. Meet the diagnostic criteria of type 1 diabetes mellitus: clinical manifestations of typical diabetes mellitus include polyphagia, polyuria, weight loss, or diabetic ketoacidosis, confirmed by blood sugar level, islet function and autoimmune antibody.
2. Existence of extrapancreatic organ damage: (1) inflammatory bowel disease, (2) impairment of renal function, (3) repeated infection of mouth, skin, anus or whole body, (4) immune hepatitis, (5) persistent chronic immune iridocyclitis, (6) immune adrenalinitis leading to adrenocortical dysfunction, (7) pituitary inflammation leading to hypophysis, (8) rheumatoid disease, (9) immune vasculitis, (10) systemic lupus erythematosus, (11) other organs besides thyroid function damage. Suffering from one or more of above diseases. Recurrence after receiving regular clinical treatment, including symptomatic treatment of organ protective drugs.
3. Gene mutation was found according to gene diagnosis: gene mutation was found by gene sequencing. Literature searches at home and abroad confirmed that the defect of the gene resulted in autoimmune or immune dysfunction, resulting in multiple organ dysfunction and poor prognosis.

Exclusion Criteria:

1. Mature and effective treatment methods are available.
2. HIV, HBV and HCV were positive.
3. A the active period of infection.
4. At the active stage of malignant tumors.
5. Combination of other fatal diseases.
6. Existence of mental and psychological diseases.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Interventional; Sequential transplantation of umbilical cord blood stem cells and islet cells

Procedure: Sequential transplantation; After successful matching of umbilical cord blood stem cells, patients will receive pretreatment and chemotherapy under protective isolation, followed by thawing and reinfusion of umbilical cord blood stem cells. Immunosuppressive agents will be used for GVHD prevention and anti-infection support will be provided after reinfusion. The status of umbilical cord blood stem cell implantation, immune reconstruction and therapeutic effect will be evaluated. Islet transplantation will be performed in those who meet the conditions. The long-term prognosis will be observed by long-term follow-up.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 3 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
HbA1c level	HbA1c level	from the completion of treatment to 3 months
HbA1c level	HbA1c level	from the completion of treatment to 6 months
HbA1c level	HbA1c level	from the completion of treatment to 9 months
HbA1c level	HbA1c level	from the completion of treatment to 12 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 6 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 9 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 12 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 15 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 18 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 21 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 24 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 27 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 30 months
Concentration of serum C-peptide	Islet function (concentration of serum C-peptide)	from the completion of treatment to 33 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 3 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 6 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 9 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 12 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 15 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 18 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 21 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 24 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 27 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 30 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 33 months
Concentration of serum insulin	Concentration of serum insulin	from the completion of treatment to 36 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 3 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 6 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 9 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 12 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 15 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 18 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 21 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 24 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 27 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 30 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 33 months
Fast blood glucose level	Fast blood glucose level	from the completion of treatment to 36 months
HbA1c level	HbA1c level	from the completion of treatment to 15 months
HbA1c level	HbA1c level	from the completion of treatment to 18 months
HbA1c level	HbA1c level	from the completion of treatment to 21 months
HbA1c level	HbA1c level	from the completion of treatment to 24 months
HbA1c level	HbA1c level	from the completion of treatment to 27 months
HbA1c level	HbA1c level	from the completion of treatment to 30 months
HbA1c level	HbA1c level	from the completion of treatment to 33 months
HbA1c level	HbA1c level	from the completion of treatment to 36 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 3 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 6 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 9 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 12 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 15 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 18 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 21 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 24 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 27 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 30 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 33 months
occurrence of infection (number of infections)	occurrence of infection (number of infections)	from the completion of treatment to 36 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 3 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 6 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 9 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 12 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 15 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 18 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 21 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 24 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 27 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 30 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 33 months
Concentration of blood immunoglobulin	Concentration of blood immunoglobulin	from the completion of treatment to 36 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 3 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 6 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 9 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 12 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 15 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 18 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 21 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 24 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 27 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 30 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 33 months
Concentration of T lymphocyte subsets	Concentration of T lymphocyte subsets	from the completion of treatment to 36 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 3 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 6 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 9 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 12 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 15 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 18 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 21 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 24 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 27 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 30 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 33 months
Concentraion of interleukin-2	Cytokines (concentraion of interleukin-2)	from the completion of treatment to 36 months
Body height	Body height	from the completion of treatment to 3 months
Body height	Body height	from the completion of treatment to 6 months
Body height	Body height	from the completion of treatment to 9 months
Body height	Body height	from the completion of treatment to 12 months
Body height	Body height	from the completion of treatment to 15 months
Body height	Body height	from the completion of treatment to 18 months
Body height	Body height	from the completion of treatment to 21 months
Body height	Body height	from the completion of treatment to 24 months
Body height	Body height	from the completion of treatment to 27 months
Body height	Body height	from the completion of treatment to 30 months
Body height	Body height	from the completion of treatment to 33 months
Body height	Body height	from the completion of treatment to 36 months
Body weight	Body weight	from the completion of treatment to 3 months
Body weight	Body weight	from the completion of treatment to 6 months
Body weight	Body weight	from the completion of treatment to 9 months
Body weight	Body weight	from the completion of treatment to 12 months
Body weight	Body weight	from the completion of treatment to 15 months
Body weight	Body weight	from the completion of treatment to 18 months
Body weight	Body weight	from the completion of treatment to 21 months
Body weight	Body weight	from the completion of treatment to 24 months
Body weight	Body weight	from the completion of treatment to 27 months
Body weight	Body weight	from the completion of treatment to 30 months
Body weight	Body weight	from the completion of treatment to 33 months
Body weight	Body weight	from the completion of treatment to 36 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 3 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 6 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 9 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 12 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 15 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 18 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 21 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 24 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 27 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 30 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 33 months
Tanner stage	Puberty change (Tanner stage)	from the completion of treatment to 36 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 3 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 6 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 9 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 12 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 15 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 18 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 21 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 24 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 27 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 30 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 33 months
Occurrence of graft versus host disease	Occurrence of graft versus host disease	from the completion of treatment to 36 months

Collaborators and Investigators

• Sponsor: Children's Hospital of Fudan University
• Investigators: Study Chair: Luo Feihong, Children's Hospital of Fudan University

Publications and helpful links

General Publications

• Cho NH, Shaw JE, Karuranga S, Huang Y, da Rocha Fernandes JD, Ohlrogge AW, Malanda B. IDF Diabetes Atlas: Global estimates of diabetes prevalence for 2017 and projections for 2045. Diabetes Res Clin Pract. 2018 Apr;138:271-281. doi: 10.1016/j.diabres.2018.02.023. Epub 2018 Feb 26.
• American Diabetes Association. (2) Classification and diagnosis of diabetes. Diabetes Care. 2015 Jan;38 Suppl:S8-S16. doi: 10.2337/dc15-S005. No abstract available.
• Fu H, Shen SX, Chen ZW, Wang JJ, Ye TT, LaPorte RE, Tajima N. Shanghai, China, has the lowest confirmed incidence of childhood diabetes in the world. Diabetes Care. 1994 Oct;17(10):1206-8. doi: 10.2337/diacare.17.10.1206.
• Zhao Z, Sun C, Wang C, Li P, Wang W, Ye J, Gu X, Wang X, Shen S, Zhi D, Lu Z, Ye R, Cheng R, Xi L, Li X, Zheng Z, Zhang M, Luo F. Rapidly rising incidence of childhood type 1 diabetes in Chinese population: epidemiology in Shanghai during 1997-2011. Acta Diabetol. 2014 Dec;51(6):947-53. doi: 10.1007/s00592-014-0590-2. Epub 2014 Apr 29.
• Daniels M, DuBose SN, Maahs DM, Beck RW, Fox LA, Gubitosi-Klug R, Laffel LM, Miller KM, Speer H, Tamborlane WV, Tansey MJ; T1D Exchange Clinic Network. Factors associated with microalbuminuria in 7,549 children and adolescents with type 1 diabetes in the T1D Exchange clinic registry. Diabetes Care. 2013 Sep;36(9):2639-45. doi: 10.2337/dc12-2192. Epub 2013 Apr 22.
• Amin R, Widmer B, Prevost AT, Schwarze P, Cooper J, Edge J, Marcovecchio L, Neil A, Dalton RN, Dunger DB. Risk of microalbuminuria and progression to macroalbuminuria in a cohort with childhood onset type 1 diabetes: prospective observational study. BMJ. 2008 Mar 29;336(7646):697-701. doi: 10.1136/bmj.39478.378241.BE. Epub 2008 Mar 18.
• Mollsten A, Svensson M, Waernbaum I, Berhan Y, Schon S, Nystrom L, Arnqvist HJ, Dahlquist G; Swedish Childhood Diabetes Study Group; Diabetes Incidence Study in Sweden; Swedish Renal Registry. Cumulative risk, age at onset, and sex-specific differences for developing end-stage renal disease in young patients with type 1 diabetes: a nationwide population-based cohort study. Diabetes. 2010 Jul;59(7):1803-8. doi: 10.2337/db09-1744. Epub 2010 Apr 27.
• Wildin RS, Smyk-Pearson S, Filipovich AH. Clinical and molecular features of the immunodysregulation, polyendocrinopathy, enteropathy, X linked (IPEX) syndrome. J Med Genet. 2002 Aug;39(8):537-45. doi: 10.1136/jmg.39.8.537.
• Uzel G, Sampaio EP, Lawrence MG, Hsu AP, Hackett M, Dorsey MJ, Noel RJ, Verbsky JW, Freeman AF, Janssen E, Bonilla FA, Pechacek J, Chandrasekaran P, Browne SK, Agharahimi A, Gharib AM, Mannurita SC, Yim JJ, Gambineri E, Torgerson T, Tran DQ, Milner JD, Holland SM. Dominant gain-of-function STAT1 mutations in FOXP3 wild-type immune dysregulation-polyendocrinopathy-enteropathy-X-linked-like syndrome. J Allergy Clin Immunol. 2013 Jun;131(6):1611-23. doi: 10.1016/j.jaci.2012.11.054. Epub 2013 Mar 25.
• Bennett CL, Christie J, Ramsdell F, Brunkow ME, Ferguson PJ, Whitesell L, Kelly TE, Saulsbury FT, Chance PF, Ochs HD. The immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX) is caused by mutations of FOXP3. Nat Genet. 2001 Jan;27(1):20-1. doi: 10.1038/83713.
• Rao A, Kamani N, Filipovich A, Lee SM, Davies SM, Dalal J, Shenoy S. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood. 2007 Jan 1;109(1):383-5. doi: 10.1182/blood-2006-05-025072. Epub 2006 Sep 21.
• van de Veerdonk FL, Plantinga TS, Hoischen A, Smeekens SP, Joosten LA, Gilissen C, Arts P, Rosentul DC, Carmichael AJ, Smits-van der Graaf CA, Kullberg BJ, van der Meer JW, Lilic D, Veltman JA, Netea MG. STAT1 mutations in autosomal dominant chronic mucocutaneous candidiasis. N Engl J Med. 2011 Jul 7;365(1):54-61. doi: 10.1056/NEJMoa1100102. Epub 2011 Jun 29.
• Toubiana J, Okada S, Hiller J, Oleastro M, Lagos Gomez M, Aldave Becerra JC, Ouachee-Chardin M, Fouyssac F, Girisha KM, Etzioni A, Van Montfrans J, Camcioglu Y, Kerns LA, Belohradsky B, Blanche S, Bousfiha A, Rodriguez-Gallego C, Meyts I, Kisand K, Reichenbach J, Renner ED, Rosenzweig S, Grimbacher B, van de Veerdonk FL, Traidl-Hoffmann C, Picard C, Marodi L, Morio T, Kobayashi M, Lilic D, Milner JD, Holland S, Casanova JL, Puel A; International STAT1 Gain-of-Function Study Group. Heterozygous STAT1 gain-of-function mutations underlie an unexpectedly broad clinical phenotype. Blood. 2016 Jun 23;127(25):3154-64. doi: 10.1182/blood-2015-11-679902. Epub 2016 Apr 25.
• Leiding JW, Okada S, Hagin D, Abinun M, Shcherbina A, Balashov DN, Kim VHD, Ovadia A, Guthery SL, Pulsipher M, Lilic D, Devlin LA, Christie S, Depner M, Fuchs S, van Royen-Kerkhof A, Lindemans C, Petrovic A, Sullivan KE, Bunin N, Kilic SS, Arpaci F, Calle-Martin O, Martinez-Martinez L, Aldave JC, Kobayashi M, Ohkawa T, Imai K, Iguchi A, Roifman CM, Gennery AR, Slatter M, Ochs HD, Morio T, Torgerson TR; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the Primary Immune Deficiency Treatment Consortium. Hematopoietic stem cell transplantation in patients with gain-of-function signal transducer and activator of transcription 1 mutations. J Allergy Clin Immunol. 2018 Feb;141(2):704-717.e5. doi: 10.1016/j.jaci.2017.03.049. Epub 2017 Jun 7.
• Niclauss N, Morel P, Berney T. Has the gap between pancreas and islet transplantation closed? Transplantation. 2014 Sep 27;98(6):593-9. doi: 10.1097/TP.0000000000000288.
• Ahearn AJ, Parekh JR, Posselt AM. Islet transplantation for Type 1 diabetes: where are we now? Expert Rev Clin Immunol. 2015 Jan;11(1):59-68. doi: 10.1586/1744666X.2015.978291. Epub 2014 Dec 2.
• Kopan C, Tucker T, Alexander M, Mohammadi MR, Pone EJ, Lakey JRT. Approaches in Immunotherapy, Regenerative Medicine, and Bioengineering for Type 1 Diabetes. Front Immunol. 2018 Jun 12;9:1354. doi: 10.3389/fimmu.2018.01354. eCollection 2018.

Study record dates

Study Major Dates

• Study Start (Actual): February 20, 2019
• Primary Completion (Estimated): December 31, 2025
• Study Completion (Estimated): December 31, 2025

Study Registration Dates

• First Submitted: January 31, 2019
• First Submitted That Met QC Criteria: February 6, 2019
• First Posted (Actual): February 8, 2019

Study Record Updates

• Last Update Posted (Actual): August 31, 2023
• Last Update Submitted That Met QC Criteria: August 29, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Keywords: Cord Blood Stem Cell Transplantation; Islets of Langerhans Transplantation
• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Immune System Diseases; Autoimmune Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 1; Immunologic Deficiency Syndromes
• Other Study ID Numbers: SeqTranUCBC&IC-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No